Study of MGTA-145 and Plerixafor in Patients With Sickle Cell Disease

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-06-24

Study Completion Date

2023-02-02

Brief Summary

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This research study is designed to investigate a new potential medicine for mobilizing stem cells and apheresis collection in patients with Sickle Cell Disease. MGTA-145, the new potential medicine, will be given with plerixafor.

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Detailed Description

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This Phase 2, multicenter, open-label study will be conducted in 2 parts (Parts A and B). Part A is intended to characterize the efficacy, safety, PK and PD of a single dose of MGTA-145 and plerixafor for HSC mobilization and apheresis collection in patients with SCD. Part B is designed to characterize the efficacy, safety, PK and PD of 2 consecutive days of dosing with MGTA-145 and plerixafor for HSC mobilization and apheresis collection in patients with SCD.

Conditions

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Sickle Cell Disease

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Part A: Single Day Dosing/Apheresis

Single dose of MGTA-145 in combination with plerixafor followed by apheresis

Group Type EXPERIMENTAL

MGTA-145

Intervention Type BIOLOGICAL

MGTA-145 will be administered as an IV infusion

Plerixafor

Intervention Type DRUG

240 µg/kg administered subcutaneously

Part B: 2-Day Dosing/Apheresis

MGTA-145 in combination with plerixafor followed by apheresis on two consecutive days

Group Type EXPERIMENTAL

MGTA-145

Intervention Type BIOLOGICAL

MGTA-145 will be administered as an IV infusion

Plerixafor

Intervention Type DRUG

240 µg/kg administered subcutaneously

Interventions

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MGTA-145

MGTA-145 will be administered as an IV infusion

Intervention Type BIOLOGICAL

Plerixafor

240 µg/kg administered subcutaneously

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Subject must be ≥18 to ≤35 years of age.
* Subject must weigh ≥30 kg.
* Subject must have a diagnosis of Sickle Cell Disease.

Exclusion Criteria

* Subject must not have had a vaso-occlusive event (VOE) requiring a visit to a healthcare facility within 30 days of screening.
* Subject must not have undergone or attempted and failed previous hematopoietic stem cell (HSC) collection.
* Subject must not have had a prior autologous or allogeneic transplantation, inclusive of gene therapy.
* Male subject must be willing or able to use a highly effective method of contraception for 3 months during and after treatment.
* Female subject must not be pregnant or breastfeeding. If sexually active, female subject must be willing or able to use a highly effective method of contraception for 3 months during and after treatment.

Minimum Eligible Age

18 Years

Maximum Eligible Age

35 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No